Intrathecal SHP611 in Subjects with Metachromatic Leukodystrophy
The primary objective of this study is to evaluate the safety and efficacy of an investigational drug known as SHP611 in individuals with a confirmed diagnosis late infantile metachromatic leukodystrophy (MLD).
Study participation lasts for about 26 months. The study will consist of a screening period of up to 28 days. All participants will receive a once weekly dose of intrathecal (IT) SHP611 for 105 weeks via an intrathecal drug delivery device (IDDD) that is implanted prior to administration of the first dose of the investigational drug. During treatment, participants will undergo assessments of gross motor function, brain imaging and quality of life.
If you are interested in learning more about this clinical trial, you may either submit our referral survey or call 267-425-0101 to speak with a study coordinator.